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PGM1-congenital disorder of glycosylation

MONDO:0013968

Also known as: CDG syndrome type It, CDG-It, CDG1T, PGM1-CDG, PGM1-congenital disorder of glycosylation, congenital disorder of glycosylation type 1t, congenital disorder of glycosylation type It, phosphoglucomutase-1 deficiency

8 clinical trials for this condition and its sub-types.

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Broader categories

Cardiovascular disorder (1051) Disease (680) Heart disorder (300) Metabolic disease (233) Musculoskeletal system disorder (207) Hereditary disease (176) Cardiomyopathy (144) Dilated cardiomyopathy (72) Muscle tissue disorder (56) Inborn errors of metabolism (45)
Trials to join now! 5 Not yet recruiting 1 Not yet finished but already full! 2
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  • Sugar supplement shows promise for rare disease in new trial

    Disease control Not yet recruiting

    This phase 2b trial tests AVTX-801, a D-galactose supplement, in 8 adults with PGM1-CDG, a rare genetic disorder affecting sugar metabolism. Participants currently on D-galactose will receive either the study drug or a placebo to see if it reduces disease-related events like low …

    Phase: PHASE2 • Sponsor: Eva Morava-Kozicz • Aim: Disease control

    Last updated Jun 27, 2026 11:01 UTC

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